Recruiting

Vamorolone

Sponsor:

Catalyst Pharmaceuticals, Inc.

Code:

NCT06564974

Conditions

Duchenne Muscular Dystrophy

Eligibility Criteria

Sex: Male

Age: 2+

Healthy Volunteers: Not accepted

Interventions

Vamorolone

Study Details

Brief summary:

The goal of this study is to collect additional information on the safety of long-term treatment with AGAMREE® and to explore long-term clinical impact of AGAMREE® on quality of life, as assessed by standardized patient-reported outcome measures (QoL questionnaires) in male patients aged 2 years and older with Duchenne muscular dystrophy (DMD).

Conditions

Duchenne Muscular Dystrophy

Study ID

NCT06564974

Start date

Sep 25, 2024

Status verified date

Sep, 2026

Completion date

Feb, 2032

Anticipated

Primary completion date

Feb, 2032

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Male

Age: 2+

Healthy Volunteers: Not accepted

Inclusion Criteria:

1. Patient or parent/legal guardian is willing and able to provide written informed consent once the nature of the registry has been explained and prior to the start of any registry-related procedures.
2. Patient and/or parent/guardian are willing and able to complete QoL questionnaires.
3. Male patients at least 2 years old.
4. Confirmed diagnosis of DMD (via genetic testing or muscle biopsy with absent dystrophin staining to anti- dystrophin antibodies 3, 1, or 2, or dystrophin immunohistochemistry or western blot).
5. Patient has a current, active prescription for, or is on, AGAMREE®.

Exclusion Criteria:

1\. Any contraindication to AGAMREE® or medical condition, which, in the opinion of the Investigator, would affect registry participation, performance, or interpretation of registry assessments.

Study Design

Enrollment

250 participants

Anticipated

Interventions and Outcome Measures

Interventions

Vamorolone

Study treatment is AGAMREE®, which is commercially available as an oral suspension.

Primary outcome measure

  • Change in Height z-score [ Time Frame: At Enrollment Visit (baseline), and at each Yearly Follow-up Visit (for up to 5 years). ]
  • Change in BMI z-score [ Time Frame: At Enrollment Visit (baseline), and each Yearly Follow-up Visit (for up to 5 years) ]
  • Change in North Star Ambulatory Assessment from baseline [ Time Frame: At Enrollment visit, and each Yearly Follow-up Visit (for up to 5 years). ]
  • Change in Performance of Upper Limb from baseline [ Time Frame: At Enrollment Visit, at each Yearly Follow-up Visit (for up to 5 years). ]
  • Tanner stage documentation [ Time Frame: At Enrollment Visit (baseline), at each Yearly Follow-up Visit (for up to 5 years). ]
  • Percentage of patients with cataract and/or glaucoma [ Time Frame: At Enrollment (baseline), and during each Yearly Follow-up Visit (for up to 5 years). ]
  • Findings from lateral spine x-ray [ Time Frame: At Enrollment Visit (baseline), and during each Yearly Follow-up Visit (for up to 5 years). ]
  • Findings from anterior-posterior/posterior-anterior X-ray [ Time Frame: At Enrollment Visit (baseline), and at each Yearly Follow-Up Visit (for up to 5 years). ]
  • Findings from hand-wrist X-ray [ Time Frame: At Enrollment Visit (baseline), and each Yearly Follow-Up Visit (for up to 5 years). ]
  • Summary statistics of bone mineral density measures assessed by dual x-ray absorptiometry [ Time Frame: At Enrollment Visit (baseline) and each Yearly Follow-Up Visit (for up to 5 years). ]
  • Body Composition Mass Measures [ Time Frame: At Enrollment Visit, each Yearly Follow-Up and End of Study/Early Termination (for approximately 5 years). ]
  • Summary statistics of body fat percentage and regional fat distribution ratios assessed by DXA [ Time Frame: At Enrollment visit, each Yearly Follow-up visit, and End of study/Early termination (for approximately 5 years). ]
  • Findings from standard of care echocardiography [ Time Frame: At Enrollment Visit, and at each Yearly Follow-up Visit (for up to 5 years). ]
  • Frequency and percentage of participants experiencing any adverse events and serious adverse events [ Time Frame: From informed consent, Enrollment visit, each Yearly follow-up visit, and End of Study/Early Termination (for up to 5 years). ]
  • Findings from cardiac magnetic resonance imaging (subset of patients taking part in the cardiac sub-study only) [ Time Frame: Enrollment Visit, and at each Yearly Follow-Up Visit (for up to 5 years). ]
  • Presence of cardiomyopathy assessed by echocardiography using transmural strain profile (only in a subset of patients taking part in the cardiac sub-study) [ Time Frame: At Enrollment Visit (baseline), and each Yearly Follow-up Visit (for up to 5 years) or Early Termination. ]

Central Contacts and Locations

Central contacts

Mark Wingertzahn, PhD

(305) 420-3200mark.wingertzahn@catalystpharma.com

Locations

Phoenix Children's Hospital

Recruiting

Phoenix, Arizona, United States, 85016

Contacts

Clara Chow, MD

602 933 0956cchow@phoenixchildrens.com

Loma Linda University Pediatric Subspecialty Clinics

Recruiting

Loma Linda, California, United States, 92354

Contacts

Monica Pizana

909-558-5830mpizana@llu.edu

Principal Investigator:

David Michelson, MD

University of California, Davis

Recruiting

Sacramento, California, United States, 95817

Contacts

Craig McDonald, MD

916 734 7041cmmcdonald@ucdavis.edu

University of Florida Clinical and Translational Science Institue

Recruiting

Gainesville, Florida, United States, 32610

Contacts

Julie Berthy, NP

352 273 8700jberthy@ufl.edu

Johns Hopkins All Children's Hospital

Recruiting

St. Petersburg, Florida, United States, 33701

Contacts

Susan Dvojack

727-898-7451sdvojac1@jhmi.edu

Principal Investigator:

Matias Lopez Chacon, MD

Cincinnati Children's Hospital Medical Center

Recruiting

Cincinnati, Ohio, United States, 45229

Contacts

Meghan McAnally, MD

513 636 9285meghan.mcanally@cchmc.org

The University of Texas Health Science Center at San Antonio

Recruiting

San Antonio, Texas, United States, 78229

Contacts

Matthew Wicklund, MD

210 450 0500wicklund@uthscsa.edu

More Information

Sponsor

Catalyst Pharmaceuticals, Inc.

Last update posted

Sep 24, 2026

Last verified

Sep, 2026

Keywords

  • Duchenne Muscular Dystrophy
  • Neuromuscular
  • AGAMREE®
  • Vamorolone
  • DMD
  • Muscular Dystrophy

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-24. This information was provided to ClinicalTrials.gov by Catalyst Pharmaceuticals, Inc. on 2026-09-24. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.